Core Insights - Capricor Therapeutics is focused on developing therapies for Duchenne cardiomyopathy and has faced regulatory challenges but is working on a clear path forward for approval [2][4] - The company has made significant progress with its exosome platform, StealthX™, which has received FDA clearance for clinical trials [4][6] - Financial results indicate a net loss for the second quarter of 2025, with revenues dropping to zero compared to the previous year [8][10] Corporate Update - Capricor aims to resubmit its Biologics License Application (BLA) for Deramiocel based on existing data, with additional data from the HOPE-3 trial expected in Q4 2025 [4][5] - The FDA has accepted all observations from the Pre-License Inspection, marking a milestone in regulatory progress [4][5] - The company is preparing for a Type A meeting with the FDA to discuss the approval pathway [4][5] Financial Performance - Cash position as of June 30, 2025, was approximately $122.8 million, down from $151.5 million at the end of 2024 [7][26] - Revenues for Q2 2025 were $0, a decrease from approximately $4.0 million in Q2 2024, and for the first half of 2025, revenues were also $0 compared to approximately $8.9 million in the first half of 2024 [8][10] - Total operating expenses for Q2 2025 were approximately $27.7 million, up from $15.6 million in Q2 2024 [9][10] Clinical Development - The HOPE-3 Phase 3 clinical trial is ongoing, with topline data expected in Q4 2025 [5][6] - The FDA has cleared the IND for the StealthX™ exosome-based vaccine, marking its first clinical entry [4][6] - Orphan Drug Designation has been granted for Deramiocel in Becker muscular dystrophy, expanding its therapeutic strategy [5][6]

Core Viewpoint - Capricor Therapeutics is advancing its lead cell therapy candidate, Deramiocel, for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD) and has scheduled a Type A meeting with the FDA to discuss its regulatory path for the Biologics License Application (BLA) [1][4][7] Financial Results and Corporate Update - The release of Capricor's financial results for the second quarter ended June 30, 2025, has been rescheduled to August 11, 2025, after market close [2][3] - A conference call and webcast will be held on the same day at 4:30 p.m. ET to review the financial results and provide a corporate update [2][3] Company Overview - Capricor Therapeutics focuses on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel being a key product candidate showing immunomodulatory and anti-fibrotic effects in muscular dystrophies [4] - The company is also utilizing its proprietary StealthX™ platform for preclinical development in areas such as vaccinology and targeted delivery of therapeutics [4] Commercialization Agreement - Capricor has entered into an exclusive agreement with Nippon Shinyaku Co., Ltd. for the commercialization and distribution of Deramiocel for DMD in the United States and Japan, pending regulatory approval [7]

Core Insights - Capricor Therapeutics, Inc. is under investigation for potential claims related to its common stock following a significant stock price drop after receiving a Complete Response Letter from the FDA regarding its lead product candidate, Deramiocel [1][2]. Company Developments - Capricor is a clinical-stage drug company focused on developing Deramiocel for treating cardiomyopathy in patients with Duchenne muscular dystrophy [1]. - The company completed its Phase 2 HOPE-2 clinical trial in 2021 [1]. - On September 24, 2024, Capricor filed a Biologics License Application (BLA) with the FDA for Deramiocel, which led to a stock price increase from $5.97 to $9.10 per share [2]. - On May 13, 2025, Capricor reported no significant deficiencies following a mid-cycle review meeting with the FDA [2]. - On July 11, 2025, Capricor announced it received a Complete Response Letter from the FDA, stating that the BLA did not meet the requirements for substantial evidence of effectiveness and required additional clinical data, resulting in a stock price drop of 33% to $7.64 per share [2]. Investor Information - Investors who experienced losses in Capricor's common stock are encouraged to contact Wolf Popper LLP for discussions regarding the investigation [3].

Core Points - Holzer & Holzer, LLC is investigating Capricor Therapeutics, Inc. for potential compliance issues with federal securities laws following the receipt of a Complete Response Letter from the FDA regarding its Biologics License Application for Deramiocel [1] - The announcement of the CRL led to a decline in Capricor's stock price [1] Company Information - Capricor Therapeutics, Inc. trades on NASDAQ under the ticker CAPR [1] - Holzer & Holzer, LLC is a law firm that specializes in representing shareholders and investors in litigation, including class action and derivative litigation [3] - The firm has a history of recovering hundreds of millions of dollars for shareholders affected by corporate misconduct since its founding in 2000 [3]

Core Viewpoint - Capricor Therapeutics Inc. received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for Deramiocel, indicating that the application cannot be approved in its current form due to insufficient evidence of effectiveness and the need for additional clinical data [1][2]. Group 1: FDA Response and Requirements - The FDA stated that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and that additional clinical data is needed [2]. - The CRL referenced outstanding items in the Chemistry, Manufacturing and Controls (CMC) section, which Capricor claims to have addressed in prior communications, but the FDA did not review these materials due to the timing of the CRL issuance [3]. Group 2: Next Steps and Company Plans - The FDA confirmed that it will restart the review clock upon resubmission and offered Capricor the opportunity to request a Type A meeting to discuss the path forward [4]. - Capricor plans to submit data from the Phase 3 HOPE-3 clinical trial to provide additional evidence of effectiveness, with topline results expected in the third quarter of 2025 [5]. Group 3: Market Reaction - Following the news, CAPR stock is down 37.4% at $7.14 during the premarket session [6].

Company Overview - Capricor Therapeutics (CAPR) shares increased by 20.4% to $9.25 in the last trading session, following a period of 25.1% loss over the past four weeks, indicating a significant recovery in stock performance [1] - The company is focused on developing Deramiocel, a cell therapy candidate aimed at treating Duchenne Muscular Dystrophy-associated cardiomyopathy [2] Regulatory Updates - Capricor provided positive regulatory updates regarding its Biologics License Application (BLA) for Deramiocel, with the FDA indicating that an Advisory Committee meeting is not required at this time [2] - The BLA is currently under Priority Review, with a target action date set for August 31, 2025, which may enhance investor confidence [2] Financial Performance Expectations - The company is expected to report a quarterly loss of $0.43 per share, reflecting a year-over-year decline of 22.9% [3] - Revenue projections for the upcoming quarter are estimated at $2.89 million, down 27.2% compared to the same quarter last year [3] Earnings Estimate Trends - The consensus EPS estimate for Capricor has remained unchanged over the last 30 days, suggesting stability in earnings expectations [4] - The stock's price movement is typically correlated with trends in earnings estimate revisions, indicating that future price strength may depend on any changes in these estimates [4] Industry Context - Capricor operates within the Zacks Medical - Products industry, which includes other companies such as Myomo, Inc. (MYO) [4] - Myomo's consensus EPS estimate has seen a significant change of -23.5% over the past month, indicating potential challenges within the same industry [5]

Core Insights - Capricor Therapeutics Inc is advancing its Biologics License Application (BLA) for Deramiocel, aimed at treating Duchenne Muscular Dystrophy (DMD)-associated cardiomyopathy [1][3] - DMD affects around 15,000 individuals in the U.S., primarily boys, due to the absence of functional dystrophin [2] Regulatory Updates - The FDA has indicated that an Advisory Committee meeting is not necessary at this time, with the BLA under Priority Review and a PDUFA target action date set for August 31, 2025 [3][4] - Capricor has been informed of a potential Advisory Committee meeting on July 30, 2025, pending FDA confirmation, with no significant issues noted during the mid-cycle review [4] Company Performance - CEO Linda Marbán stated that the company has successfully met all key regulatory milestones, including a pre-license inspection and mid-cycle review [5] - Capricor recently presented four-year data from its HOPE-2 Open-Label Extension study, showcasing one of the longest treatment datasets in DMD, focusing on cardiac and skeletal muscle function [6] Market Reaction - Following these updates, CAPR stock experienced a 20% increase, reaching $9.22 [6]

Core Insights - Capricor Therapeutics is advancing its lead cell therapy candidate, Deramiocel, for the treatment of Duchenne Muscular Dystrophy (DMD) associated cardiomyopathy, with a Biologics License Application (BLA) under Priority Review by the FDA, targeting an action date of August 31, 2025 [1][2][3] Regulatory Updates - The FDA has indicated that an Advisory Committee meeting is not required at this time, and the BLA remains under Priority Review [2][7] - A late-cycle review meeting is scheduled for mid-July 2025 [7] Clinical Data - Capricor presented four-year data from its HOPE-2 Open-Label Extension study at the 2025 Parent Project Muscular Dystrophy Conference, showing sustained cardiac function and clinical benefits from long-term Deramiocel treatment [3][7] Product Information - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) that have demonstrated immunomodulatory and anti-fibrotic actions in preserving cardiac and skeletal muscle function in DMD [5][8] - The product has received multiple designations, including Orphan Drug Designation and Regenerative Medicine Advanced Therapy designation from the FDA [6][10] Market and Collaboration - Capricor has entered into an exclusive commercialization agreement for Deramiocel in the United States and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [10]

Core Insights - Capricor Therapeutics announced positive four-year safety and efficacy results for Deramiocel, its lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD) [1][3] - The findings will be presented at the PPMD 2025 Annual Conference, highlighting the importance of addressing both cardiac and skeletal muscle functions in DMD treatment [1][4] Efficacy Results - After four years of treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline, indicating clinical benefit, especially in patients with baseline LVEF >45% [2] - The treatment also slowed skeletal muscle disease progression, with a smaller average decline in Performance of the Upper Limb (PUL v2.0) in the fourth year (0.6 points) compared to the first year (1.8 points) [3][7] Safety Profile - Deramiocel maintained a favorable safety profile throughout the study, reinforcing its potential as a therapeutic option for DMD [3][7] Regulatory Progress - Capricor is in the process of obtaining regulatory approval for Deramiocel, with its Biologics License Application (BLA) under priority review and no evidence of delays in discussions with the FDA [4][10] Study Background - The HOPE-2 study was a randomized, double-blind, placebo-controlled Phase 2 trial, with patients receiving intravenous infusions of Deramiocel (150 million cells) every three months [5] - Following the study, all patients entered a treatment gap phase before enrolling in the HOPE-2 Open-Label Extension (OLE) study, which continues to monitor safety and efficacy [5] About Duchenne Muscular Dystrophy - DMD is a severe genetic disorder affecting approximately 15,000 individuals in the U.S., characterized by progressive muscle degeneration and leading to cardiomyopathy, which is a major cause of mortality [6][9] About Deramiocel - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) known for their immunomodulatory and anti-fibrotic properties, which may help preserve cardiac and skeletal muscle function in DMD [8][10] - The therapy has received multiple designations from regulatory agencies, including Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) designation [9]

Core Viewpoint - Capricor Therapeutics has received Orphan Drug Designation from the FDA for its lead cell therapy candidate, Deramiocel, for the treatment of Becker Muscular Dystrophy, enhancing its strategic position in the neuromuscular disease market [1][3][7] Company Overview - Capricor Therapeutics is a biotechnology company focused on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel as its lead product candidate [9] - The company is advancing a fully integrated platform targeting cardiac and skeletal complications associated with muscular dystrophy [1][9] Product Details - Deramiocel (CAP-1002) is an allogeneic cell therapy derived from cardiosphere-derived cells (CDCs), which have shown immunomodulatory and anti-fibrotic effects in preserving muscle function in dystrophies [6][9] - The therapy is currently in late-stage development for Duchenne Muscular Dystrophy (DMD) and has received multiple designations from regulatory bodies, including Orphan Drug Designation for both DMD and Becker Muscular Dystrophy [8][11] Disease Context - Becker Muscular Dystrophy (BMD) is a progressive neuromuscular disorder affecting approximately 5,000 individuals in the U.S., characterized by slower disease progression compared to Duchenne Muscular Dystrophy (DMD) [5] - Both BMD and DMD are caused by mutations in the dystrophin gene, leading to significant muscle deterioration and serious cardiac complications [2][4][5] Regulatory Milestones - Capricor has successfully completed its Pre-License Inspection, a key regulatory milestone for its Biologics License Application (BLA) for DMD, with a target action date set for August 31, 2025 [3][7] - The company is preparing for the potential commercial launch of Deramiocel, aiming to deliver meaningful benefits to patients with both DMD and BMD [3][7]